More information on this, from Oxford Biomedica's website

ProSavin®

ProSavin® is a gene-based treatment for Parkinson's disease, a progressive movement disorder caused by the degeneration of dopamine producing nerve cells in the brain. In Parkinson's disease, there is degeneration of the cells in the brain that produce dopamine. ProSavin® uses the Company's LentiVector® gene delivery technology to deliver the genes for three enzymes that are required for the synthesis of dopamine. The product is administered locally to the region of the brain called the striatum, converting cells into a replacement dopamine factory within the brain, thus replacing the patient's own lost source of the neurotransmitter.
Encouraging Phase I/II results

ProSavin® is being evaluated in a Phase I/II trial in patients with mid-stage Parkinson's disease. The first stage of the study is an open-label dose escalation of ProSavin® in cohorts of three patients. The first two dose levels (1x and 2x) were safe, well-tolerated and showed promising evidence of efficacy with a maximum improvement in motor function of up to 56% at the 2x dose. If these results are confirmed in placebo-controlled studies, ProSavin® would represent a significant advancement to current treatment options.
Enhanced administration

Results from the third cohort of patients treated using a new infusion technique reduced the surgical delivery time by 50% at the 2x dose. The enhanced technique requires fewer needle tracks into the brain and is capable of delivering higher doses of ProSavin® compared to the previous administration procedure. Following approval from the Data Monitoring Committee in December 2010, Oxford BioMedica plans to proceed to a 5x dose level in early 2011, with the aim of advancing ProSavin® into a randomised Phase II study in 2012.

Market opportunity

Parkinson’s disease currently affects 4.1 million patients globally which is projected to rise to 8.7 million by 2030. A patient with Parkinson’s disease progressively loses the ability to make the neurotransmitter dopamine, the mediator of the control of movement. There is no cure for Parkinson’s disease, and no treatment can prevent the disease from progressing, however current treatments can usually ease symptoms. The current treatment market for Parkinson’s disease is approximately US$3.5 billion (source: Datamonitor). ProSavin® has the potential to address an unmet medical need in Parkinson’s disease, offering long-lasting benefit from a single administration with an excellent safety profile. The product could also reduce the social care burden that is associated with mid to late-stage disease.

Oxford BioMedica plc
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The Oxford Science Park, Oxford,
OX4 4GA United Kingdom


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